High event-free survival rate with minimum-dose-anthracycline treatment in childhood acute promyelocytic leukaemia: a nationwide prospective study by the Japanese Paediatric Leukaemia/Lymphoma Study Group

Hiroyuki Takahashi, Tomoyuki Watanabe, Akitoshi Kinoshita, Yuki Yuza, Hiroshi Moritake, Kiminori Terui, Shotaro Iwamoto, Hideki Nakayama, Akira Shimada, Kazuko Kudo, Tomohiko Taki, Miharu Yabe, Hiromichi Matsushita, Yuka Yamashita, Kazutoshi Koike, Atsushi Ogawa, Yoshiyuki Kosaka, Daisuke Tomizawa, Takashi Taga, Akiko M. SaitoKeizo Horibe, Tatsutoshi Nakahata, Hayato Miyachi, Akio Tawa, Souichi Adachi

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10 Citations (Scopus)

Abstract

We evaluated the efficacy of treatment using reduced cumulative doses of anthracyclines in children with acute promyelocytic leukaemia (APL) in the Japanese Paediatric Leukaemia/Lymphoma Study Group AML-P05 study. All patients received two and three subsequent courses of induction and consolidation chemotherapy respectively, consisting of all-trans retinoic acid (ATRA), cytarabine and anthracyclines, followed by maintenance therapy with ATRA. Notably, a single administration of anthracyclines was introduced in the second induction and all consolidation therapies to minimize total doses of anthracycline. The 3-year event-free (EFS) and overall survival rates for 43 eligible children were 83·6% [95% confidence interval (CI): 68·6–91·8%] and 90·7% (95% CI: 77·1–96·4%), respectively. Although two patients died of intracranial haemorrhage or infection during induction phases, no cardiac adverse events or treatment-related deaths were observed during subsequent phases. Patients not displaying M1 marrow after the first induction therapy, or those under 5 years of age at diagnosis, showed inferior outcomes (3-year EFS rate; 33·3% (95% CI: 19·3–67·6%) and 54·6% (95% CI: 22·9–78·0%), respectively). In conclusion, a single administration of anthracycline during each consolidation phase was sufficient for treating childhood APL. In younger children, however, conventional ATRA and chemotherapy may be insufficient so that alternative therapies should be considered.

Original languageEnglish
Pages (from-to)437-443
Number of pages7
JournalBritish Journal of Haematology
Volume174
Issue number3
DOIs
Publication statusPublished - 01-08-2016

All Science Journal Classification (ASJC) codes

  • Hematology

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